An Ocular Therapeutix drug developed for the wet form of age-related macular degeneration was better at helping patients maintain vision compared to a blockbuster Regeneron Pharmaceuticals drug that is a standard treatment for the vision-loss disorder, Phase 3 results that also show the therapy’s potential to offer patients less frequent eye injections to treat the vision-loss disorder.
A second Phase 3 test of the Ocular drug, Axpaxli, is ongoing. But based on the preliminary results reported from the first test, the company said it plans to meet with the FDA to discuss plans for a regulatory submission.
In the wet form of age-related macular degeneration, or AMD, abnormal blood vessel growth underneath the retina leaks fluid that affects the macula, the central part of the retina. Axpaxli’s main pharmaceutical ingredient is axitinib, a small molecule tyrosine kinase inhibitor intended to stop this blood vessel growth. The drug employs a bioresorbable hydrogel technology that can time the release of a drug anywhere from days to months. Ocular is aiming for a dosing interval of six months or longer.
The Axpaxli data reported Tuesday are from a Phase 3 test that evaluated a single dose of Ocular’s drug compared to a single dose of Regeneron’s Eylea. Those doses followed an eight-week loading portion of the study in which all 344 newly diagnosed wet AMD study participants received two injections of Eylea.
Results show that at week 36, 74.1% of patients in the Axpaxli arm maintained vision compared to 55.8% of participants in the Eylea group, a statistically significant outcome that met the trial’s primary endpoint. The study remained masked to assess durability of the therapy. At week 52, 65.9% of patients in the Axpaxli arm maintained vision compared to 44.2% of those in the comparator group.
To date, Ocular said the study drug has been generally well tolerated with no eye or systemic serious adverse events reported. At the 52-week mark, participants were re-dosed with the treatments that they initially received and will be re-dosed again at week 76. Masking will continue and the study will follow patients through week 104. The company plans to present more detailed results from the study so far during the Macula Society Annual Meeting in San Diego next week.
Despite the study meeting its goals, Ocular’s stock price opened Tuesday at $6.41, down more than 27% from Friday’s closing price. In a research note, William Blair analyst Lachlan Hanbury-Brown said the difference in the treatment effect between the two arms was less than what investors were looking for. Even so, he said the investment bank believes these results support FDA approval. He noted that the results for the Axpaxli arm were in line with expectations. The smaller difference in treatment effect was driven by the control arm outperforming expectations rather than Axpaxli underperforming.
The second Phase 3 test of Axpaxli is expected to yield preliminary data in the first quarter of next year. Hanbury-Brown said the data so far have positive readthrough to this study as well as a separate Phase 3 program evaluating the drug in non-proliferative diabetic retinopathy.
“While additional data will be useful in fully characterizing the profile of Axpaxli, we believe these data de-risk its development program in wet AMD and likely also the HELIOS program in diabetic eye disease,” Hanbury-Brown said.
Less frequent dosing will be important for Ocular as it aims to break into a wet AMD field dominated by Regeneron’s Eylea (marketed by Bayer outside the U.S.) and Roche’s Vabysmo. Eylea is administered as an eye injection every four weeks for the first three months and then every eight weeks for maintenance dosing. Vabysmo is administered every four weeks for four months, then every four to eight weeks, depending on the patient’s response to the therapy. While both drugs block VEGF proteins that promote blood vessel growth, Vabysmo pairs that mechanism of action with inhibition of a second protein called Ang-2.
Ocular has competition in applying tyrosine kinase inhibition to wet AMD. The main pharmaceutical ingredient in EyePoint Pharmaceuticals drug candidate Duravyu is vorolanib, a tyrosine kinase inhibitor delivered with a proprietary technology that enables sustained delivery of a therapy to the eye. Two Phase 3 studies are ongoing evaluating dosing every six months. EyePoint has said it expects preliminary data from the first study in mid-2026.
Photo: Karen Bleier/AFP, via Getty Images
